Alex­ion’s Soliris wows in rare dis­ease PhI­II tri­al, open­ing up a new fran­chise worth $500M-$700M

Three years af­ter it launched, Alex­ion $ALXN says that the Phase III study of its cash cow Soliris for rare cas­es of re­laps­ing neu­romyelitis op­ti­ca spec­trum dis­or­der scored a clear and con­vinc­ing win, set­ting up some near term ap­pli­ca­tions that one promi­nent an­a­lyst ex­pects could be worth up to $700 mil­lion a year in new mon­ey.

Lud­wig Hantson

The dis­ease, NMOSD, is of­ten con­fused with mul­ti­ple scle­ro­sis as the im­mune sys­tem at­tacks healthy tis­sue in the spine and eyes, re­spon­si­ble for blind­ing and some­times killing pa­tients. The drug in­hibits the com­ple­ment sys­tem, which in­ves­ti­ga­tors be­lieve plays a key role in the au­toim­mune dis­or­der.

The Phase III da­ta were stel­lar. 

In­ves­ti­ga­tors re­port­ed that the drug “re­duced the risk of NMOSD re­lapse by 94.2% com­pared to place­bo (p < 0.0001). At 48 weeks, 97.9% of pa­tients re­ceiv­ing Soliris were free of re­lapse com­pared to 63.2% of pa­tients re­ceiv­ing place­bo.”

Ge­of­frey Porges, Leerink

Those num­bers had Leerink’s Ge­of­frey Porges singing the drug’s prais­es Mon­day morn­ing. He es­ti­mat­ed that this in­di­ca­tion alone could be worth an added $500 mil­lion to $700 mil­lion a year for the fran­chise, which is one of the most ex­pen­sive drugs in the world. Added Porges:

Though Soliris had suc­cess­ful re­sults in phase I, we be­lieved a pos­i­tive phase III out­come was not ap­pre­ci­at­ed by con­sen­sus giv­en sig­nif­i­cant tri­al de­sign changes from phase I to phase III that added un­cer­tain­ty about the phase III tri­al. We would ex­pect reg­u­la­to­ry fil­ings to be com­plet­ed by year-end and for the drug to be avail­able at least in the US by next year.

In­vestors liked the tone to­day, bid­ding up shares by 8% in ear­ly trad­ing.

Alex­ion, un­der new man­age­ment af­ter CEO Lud­wig Hantson took the helm last year, will now swift­ly spread out around the world to hunt up some key ap­provals in the US, Eu­rope and Japan. Hantson has been hus­tling a Soliris suc­ces­sor, ALXN-1210, to the mar­ket as he an­gles to build a pipeline of new drugs. In the mean­time, though, the biotech re­mains de­pen­dent on Soliris, with ri­vals hop­ing to snatch the crown away.

Michael Levy

“The pri­ma­ry goal in treat­ing NMOSD is re­lapse pre­ven­tion as each re­lapse fur­ther in­creas­es dis­abil­i­ty, which makes this dis­ease so dev­as­tat­ing. For decades, we have been hop­ing for a ther­a­py that can pre­vent re­lapse and sub­se­quent ac­cu­mu­la­tion of dis­abil­i­ty by ad­dress­ing a crit­i­cal un­der­ly­ing cause of the dis­ease,” said Michael Levy, as­so­ciate pro­fes­sor at Johns Hop­kins Uni­ver­si­ty.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

CDC’s Robert Redford, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA, ques­tions need to length­en process

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary deliberately muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

News brief­ing: HHS chas­tis­es Eli Lil­ly for end­ing some dis­counts in a pan­dem­ic; Gilead to pay $97 mil­lion to set­tle kick­back al­le­ga­tions

Gilead agreed to pay $97 million to settle allegations that it illegally used a charity to pay thousands of Medicare patients’ co-payments for its pulmonary arterial hypertension drug Letairis.

The US government claimed that between June 2007 and December 2010, Gilead $GILD used the Caring Voice Coalition as a conduit to cover the costs of just its own drug. The alleged behavior goes against the False Claims Act.

Covid-19 roundup: Brazil, In­done­sia, Turkey could get Sino­vac vac­cine at same time as Chi­na; Small biotech touts big suc­cess

Shooting for its first OKs by year-end, China’s Sinovac said the countries that are currently hosting its Phase III trial could be first in line to receive its Covid-19 vaccine, according to the CEO.

That could mean Brazil, Indonesia and Turkey — all battling surging cases and deaths — will get the first batch of coronavirus shots at the same time as China, which is in turn outlining a similar timeline as the US.

On­ly five months af­ter a Se­ries A launch, Taysha goes pub­lic with $157M IPO

As has been the trend in 2020, Taysha Gene Therapies has become the latest biotech to make a quick ascent from a small, privately-funded company to enjoying its very own Nasdaq ticker.

The Dallas-based biotech raised $157 million for its IPO after pricing shares at $20 apiece Thursday, the high-point of its expected range. Initially pegging $100 million in financing, Taysha offered a little less than 8 million shares and will trade under the $TSHA symbol.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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